In this issue of Hematology/Oncology Clinics, guest editors Drs. Sung-Yun Pai and Nirali N. Shah bring their considerable expertise to the topic of Gene-Based Therapies for Pediatric Blood Diseases. Top experts in the field cover key topics such as CAR T-cell therapy:: current status; engineered T cells; NK-cell therapy; hemoglobinopathies:: beta-thalassemia, sickle cell disease; hemophilia A/B; primary immunodeficiencies; and more.
Evolution of Gene Therapy, Historical PerspectiveA Curative DNA Code for Hematopoietic Defects: Novel Cell Therapies for Monogenic Diseases of the Blood and Immune SystemAdeno-Associated Virus Vector Design-Moving the Adeno-Associated Virus to a Bioengineered Therapeutic NanoparticleRegulatory Aspects of Gene Therapy: The Regulatory Life CycleChimeric Antigen Receptor T-cell Therapy: Current Status and Clinical Outcomes in Pediatric Hematologic MalignanciesGenome-Edited T Cell TherapiesGene-Based Natural Killer Cell Therapies for the Treatment of Pediatric Hematologic MalignanciesGene Therapy for Hemoglobinopathies: Beta-Thalassemia, Sickle Cell DiseaseHemophilia A/BGene Therapy for Inborn Errors of Immunity: Severe Combined ImmunodeficienciesGenes as Medicine: The Development of Gene Therapies for Inborn Errors of ImmunityGene Therapy for Pediatric Neurologic DiseaseEx Vivo and In Vivo Gene Therapy for Mucopolysaccharidoses: State of the Art
Comments (0)
Your review appreciation cannot be sent
Report comment
Are you sure that you want to report this comment?
Report sent
Your report has been submitted and will be considered by a moderator.